A new methodology, based on high resolution liquid chromatography with light scatterin detector, is applied to analyze Hydroxypropyl-beta-Cyclodextrin (HPβCD) in urine samples of a child affected by Niemann-Pick Type C disease. The treatment not only stopped disease progression, but has also increased the life expectancy and quality of our patient. The pharmacokinetic of HPβCD in the patient was studied with a 92.8% of HPβCD recovered. At 88 h, no HPβCD was found in the urine. During the treatment, HPβCD has not shown toxicity. Before application of the new treatment, injections were given every two weeks but, we have demonstrated that this can be increased to every four days.

一种基于高分辨率液相色谱和光散射检测器的新方法被用于分析尼曼-匹克型C型疾病患儿尿液样品中的羟丙基-β-环糊精（HPβCD）。该治疗不仅停止了疾病的进展，而且还提高了我们患者的预期寿命和质量。研究了HPβCD在患者体内的药代动力学，回收了92.8％的HPβCD。在88小时时，尿液中未发现HPβCD。在治疗过程中，HPβCD未显示毒性。在应用新疗法之前，每两周注射一次，但是我们证明可以增加到每四天注射一次。